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BR112015024760B8 - Antisense oligonucleotide, pharmaceutical composition and use thereof in the prevention and/or treatment of an ophthalmic disease - Google Patents

Antisense oligonucleotide, pharmaceutical composition and use thereof in the prevention and/or treatment of an ophthalmic disease

Info

Publication number
BR112015024760B8
BR112015024760B8 BR112015024760A BR112015024760A BR112015024760B8 BR 112015024760 B8 BR112015024760 B8 BR 112015024760B8 BR 112015024760 A BR112015024760 A BR 112015024760A BR 112015024760 A BR112015024760 A BR 112015024760A BR 112015024760 B8 BR112015024760 B8 BR 112015024760B8
Authority
BR
Brazil
Prior art keywords
prevention
treatment
oligonucleotide
tgf
pharmaceutical composition
Prior art date
Application number
BR112015024760A
Other languages
Portuguese (pt)
Other versions
BR112015024760B1 (en
BR112015024760A2 (en
Inventor
Eugen Leo
Eugen Uhlmann
Frank Jaschinski
Michel Janicot
Original Assignee
Isarna Therapeutics Gmbh
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Isarna Therapeutics Gmbh filed Critical Isarna Therapeutics Gmbh
Publication of BR112015024760A2 publication Critical patent/BR112015024760A2/en
Publication of BR112015024760B1 publication Critical patent/BR112015024760B1/en
Publication of BR112015024760B8 publication Critical patent/BR112015024760B8/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/12—Ophthalmic agents for cataracts
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

oligonucleotídeo gf-beta modificado, seu uso na prevenção e/ou tratamento de uma doença oftálmica e composição farmacêutica. a presente invenção refere-se a um oligonucleotídeo consistindo em 10 a 20 nucleotídeos de regiões selecionadas da sequência de ácido nucleico de tgf-beta1, tgf-beta2 ou tgf-beta3, que compreende nucleotídeos modificados, tais como nucleotídeos modificados de lna, ena, óxido de polialquileno-, 2'-flúor, 2'-o- metóxi e/ou 2'-o-metil. a invenção se relaciona adicionalmente a composições farmacêuticas compreendendo tal oligonucleotídeo, no qual a composição ou o oligonucleotídeo é usada em um método para a prevenção e/ou tratamento de glaucoma, opacificação capsular posterior, olho seco, síndrome de marfan ou de loeys-dietz, riboblastoma, coroido-carcinoma, degeneração macular, tal como degeneração macular relacionada à idade, edema macular diabético, ou catarata.modified gf-beta oligonucleotide, its use in the prevention and/or treatment of an ophthalmic disease and pharmaceutical composition. the present invention relates to an oligonucleotide consisting of 10 to 20 nucleotides from selected regions of the tgf-beta1, tgf-beta2 or tgf-beta3 nucleic acid sequence, which comprises modified nucleotides such as modified lna, ena, polyalkylene-, 2'-fluorine, 2'-o-methoxy and/or 2'-o-methyl oxide. the invention further relates to pharmaceutical compositions comprising such an oligonucleotide, in which the composition or oligonucleotide is used in a method for the prevention and/or treatment of glaucoma, posterior capsular opacification, dry eye, marfan or loeys-dietz syndrome, riboblastoma, choroido-carcinoma, macular degeneration, such as age-related macular degeneration, diabetic macular edema, or cataract.

BR112015024760A 2013-03-27 2014-03-27 Antisense oligonucleotide, pharmaceutical composition and use thereof in the prevention and/or treatment of an ophthalmic disease BR112015024760B8 (en)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
EP13161474 2013-03-27
EP13161474.5 2013-03-27
EP13173078.0 2013-06-20
EP13173078 2013-06-20
EP13199831 2013-12-30
EP13199831.2 2013-12-30
EP13199826.2 2013-12-30
EP13199826 2013-12-30
EP13199838 2013-12-30
EP13199838.7 2013-12-30
PCT/EP2014/056222 WO2014154836A2 (en) 2013-03-27 2014-03-27 Modified tgf-beta oligonucleotide for use in a method of preventing and/or treating an ophthalmic disease

Publications (3)

Publication Number Publication Date
BR112015024760A2 BR112015024760A2 (en) 2017-10-24
BR112015024760B1 BR112015024760B1 (en) 2021-09-21
BR112015024760B8 true BR112015024760B8 (en) 2022-06-21

Family

ID=50440642

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112015024760A BR112015024760B8 (en) 2013-03-27 2014-03-27 Antisense oligonucleotide, pharmaceutical composition and use thereof in the prevention and/or treatment of an ophthalmic disease

Country Status (17)

Country Link
US (3) US9688988B2 (en)
EP (1) EP2978846B1 (en)
JP (1) JP6475226B2 (en)
KR (1) KR102094572B1 (en)
CN (1) CN105283551B (en)
BR (1) BR112015024760B8 (en)
CA (1) CA2908101C (en)
DK (1) DK2978846T3 (en)
EA (1) EA201591594A1 (en)
ES (1) ES2784213T3 (en)
HK (1) HK1221254A1 (en)
HU (1) HUE049246T2 (en)
LT (1) LT2978846T (en)
PL (1) PL2978846T4 (en)
PT (1) PT2978846T (en)
SI (1) SI2978846T1 (en)
WO (1) WO2014154836A2 (en)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018009895A1 (en) * 2016-07-08 2018-01-11 Autotelic Llc Methods for trabedersen dosing by auc
EP3538655A1 (en) * 2016-11-11 2019-09-18 ISARNA Therapeutics GmbH Tgf-beta oligonucleotide for use in treatment of ophthalmic diseases
EP3596117A4 (en) 2017-03-17 2021-01-13 The Johns Hopkins University Targeted epigenetic therapy against distal regulatory element of tgfb2 expression
CN113018508A (en) * 2021-03-15 2021-06-25 西安交通大学医学院第一附属医院 Surface-modified artificial lens and preparation method thereof
KR102549208B1 (en) * 2021-04-08 2023-06-30 연세대학교 산학협력단 Long Noncoding RNA Implicated in Cardiovascular Disease and Use Thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1008649B1 (en) 1993-04-30 2003-03-26 Biognostik Gesellschaft für biomolekulare Diagnostik mbH Antisense-oligonucleotides for the treatment of immuno-suppressive effects of transforming growth factor-b2(TGF-b2)
DE69429617T2 (en) 1993-04-30 2002-08-22 Biognostik Gesellschaft Fuer Biomolekulare Diagnostik Mbh ANTISENSE OLIGONUCLEOTIDES FOR TREATING THE IMMUNE SUPPRESSIVE EFFECT OF THE TRANSFORMING GROWTH FACTOR BETA1 (TGF-BETA1)
US20040006030A1 (en) 2002-07-02 2004-01-08 Isis Pharmaceuticals Inc. Antisense modulation of TGF-beta 2 expression
EP1722823A2 (en) * 2004-02-27 2006-11-22 Antisense Pharma GmbH Pharmaceutical composition
EP1568383A3 (en) 2004-02-27 2005-11-16 Antisense Pharma GmbH Use of an oligonucleotide or its active derivative for the preparation of a pharmaceutical composition for inhibiting the formation of metastases in cancer treatment
EP1935428A1 (en) 2006-12-22 2008-06-25 Antisense Pharma GmbH Oligonucleotide-polymer conjugates
CA2802089A1 (en) * 2010-06-11 2011-12-15 Antisense Pharma Gmbh Method for selective oligonucleotide modification
EP2453017A1 (en) * 2010-11-12 2012-05-16 Antisense Pharma GmbH Oligonucleotides for use in prophylaxis and/or treatment of TGF-beta1 and TGF-beta2, TGF-beta2 and TGF-beta3, TGF-beta1 and TGF-beta3, or TGF-beta1, TGF-beta2, and TGF-beta3 mRNA overexpressing diseases
US10058623B2 (en) 2012-05-16 2018-08-28 Translate Bio Ma, Inc. Compositions and methods for modulating UTRN expression

Also Published As

Publication number Publication date
US20160040167A1 (en) 2016-02-11
LT2978846T (en) 2020-04-10
JP6475226B2 (en) 2019-02-27
US20190144865A1 (en) 2019-05-16
WO2014154836A3 (en) 2014-11-20
EP2978846A2 (en) 2016-02-03
PL2978846T3 (en) 2020-09-21
SI2978846T1 (en) 2020-07-31
PT2978846T (en) 2020-04-21
US20170314025A1 (en) 2017-11-02
BR112015024760B1 (en) 2021-09-21
DK2978846T3 (en) 2020-04-06
KR20150140702A (en) 2015-12-16
CN105283551A (en) 2016-01-27
EA201591594A1 (en) 2016-05-31
CN105283551B (en) 2018-11-06
US9688988B2 (en) 2017-06-27
WO2014154836A2 (en) 2014-10-02
BR112015024760A2 (en) 2017-10-24
KR102094572B1 (en) 2020-03-30
PL2978846T4 (en) 2020-09-21
HUE049246T2 (en) 2020-09-28
JP2016519573A (en) 2016-07-07
EP2978846B1 (en) 2019-12-25
ES2784213T3 (en) 2020-09-23
US10125368B2 (en) 2018-11-13
CA2908101A1 (en) 2014-10-02
CA2908101C (en) 2021-06-22
HK1221254A1 (en) 2017-05-26

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Legal Events

Date Code Title Description
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B06A Patent application procedure suspended [chapter 6.1 patent gazette]
B15K Others concerning applications: alteration of classification

Free format text: AS CLASSIFICACOES ANTERIORES ERAM: C12N 15/113 , A61K 31/713 , C07H 21/02 , A61P 27/02 , A61P 35/00

Ipc: C12N 15/113 (2010.01), C07H 21/02 (2006.01), A61K

B09A Decision: intention to grant [chapter 9.1 patent gazette]
B16A Patent or certificate of addition of invention granted [chapter 16.1 patent gazette]

Free format text: PRAZO DE VALIDADE: 20 (VINTE) ANOS CONTADOS A PARTIR DE 27/03/2014, OBSERVADAS AS CONDICOES LEGAIS.

B16C Correction of notification of the grant [chapter 16.3 patent gazette]

Free format text: REF. RPI 2646 DE 21/09/2021 QUANTO AO INVENTOR POR ERRO MATERIAL DO USUARIO.